Cereno Scientifics leading drug candidate, CS1, is an HDAC inhibitor that acts through epigenetic modulation. CS1 is being developed as a First-in-Class-treatment for the rare disease pulmonary arterial hypertension (PAH).
The company has completed a Phase IIa study with CS1 in PAH, showing a favorable safety profile and signals of disease-modifying effects. In the first quarter, additional data from the Phase IIa study with CS1 were shared after the clinical study report was completed. The results showed sustained improvements in REVEAL 2.0 risk score, functional level and patient-reported quality of life – early signals of long-term therapeutic effect. In addition, further data analysis also showed early signs of improved right ventricular function in the heart, which is a predictive factor for survival in PAH.
Focus on Expanded Access Program and preparations for Phase IIb
CS1 has been approved by the FDA for an Expanded Access Program (EAP) as an extension of the Phase IIa study, which allows patients who have completed the study to have access to CS1. The Expanded Access Program has continued as planned and in February a substudy was initiated where Fluiddas Imaging techniques are used to analyze structural changes in the blood vessels of the lungs during long-term treatment with CS1.
At the same time, preparations are underway for the next stage of development. In April, a Type C meeting was held with FDA, where agreement was reached on the design of an upcoming Phase IIb study – a crucial step in taking CS1 further towards broader clinical validation.
Milestone with CS014
In parallel, Cereno Scientific has made clinical progress with its second HDAC inhibitor, CS014. The first part of the Phase I study with the candidate was completed in February without safety concerns. The second part (multiple ascending dose, MAD) proceeded as planned. On April 16, the company announced that the entire Phase I study has been completed and that top-line results are expected in June 2025. According to the company, CS014 has a multimodal mechanism of action with the potential to address the underlying pathophysiology of rare cardiovascular diseases and lung diseases with high medical needs. The initial focus is the rare disease idiopathic pulmonary fibrosis (IPF).
Strengthened patent protection
During the first quarter of the year, the IP protection for CS1 was also strengthened. On March 17, a new patent was granted in the US for CS1's second patent family. Two additional patent applications have been submitted, with the aim of expanding the intellectual property protection around the drug candidate. These new applications strengthen the conditions for long-term market exclusivity for CS1 in PAH, potentially until 2045.
Company cash
According to the Q1 report, the company has mainly invested in the ongoing Expanded Access program with CS1, the Phase I study evaluating the safety and tolerability of CS014, toxicology studies for CS014 in preparation for Phase II, and the preclinical program with the company's third candidate, CS585.
Cash flow from operating activities was SEK -24,3 million during the quarter, and at the end of the quarter, the company's cash amounted to SEK 77,0 million.
– With two HDAC inhibitor programs in clinical development, a preclinical candidate being advanced in the program, a growing patent portfolio, and strong scientific and regulatory momentum, Cereno Scientific is well positioned to continue its journey towards becoming a leading player in epigenetic modulating therapies for rare cardiovascular and lung diseases, concludes Sten R. Sörensen his CEO's words in the report.